The nation’s capital is home to dozens of organizations that advocate for patients with rare diseases, ranging from the ALS Association and the Cystic Fibrosis Foundation to the Global Liver Institute and the National Organization for Rare Disorders (NORD).

Yet the nonprofit Alliance for Regenerative Medicine (ARM)—while not a patient advocacy group itself—stands out for its singular mission: to support the development of safe and effective gene therapies, cell therapies, tissue engineering, and other regenerative therapies that could benefit rare disease patients worldwide.

As such, ARM speaks for some 425 member institutions across 25 countries. Half of these members are corporations whose annual revenues exceed $50 million. The other half consists of smaller companies, nonprofit organizations, academic entities, and patient groups.

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Janet Lynch Lambert, CEO of the Alliance for Regenerative Medicine. Credit: Larry Luxner

ARM’s timing couldn’t be better, said the organization’s CEO, Janet Lynch Lambert.

“We’re in a golden age of new medicines,” she told Rare Disease Advisor in an interview following the 2022 World Orphan Drug Congress in Boston, Massachusetts. “We have new categories of therapies that stand to provide treatments, and potentially, even cures to patients suffering from a wide variety of indications. All these organizations are working together to solve the problems that stand between where we are today and where we want to get.”

Lambert will step down in September after 5 years at the helm of ARM. Her replacement is biotech executive Timothy D. Hunt, who was most recently chief culture and corporate affairs officer at Xilio Therapeutics, which develops tumor-selective immuno-oncology therapies for cancer patients.

Lambert said ARM differs from groups such as NORD and the EveryLife Foundation for Rare Diseases in that “these are fantastic organizations that work very effectively to represent the patient voice in policy deliberations, where ARM represents, in large measure, the developers of therapies. We’re a little bit more attuned to what are the commercial challenges of actually developing a therapy and getting it through a regulatory authority, whether that’s in the United States or Europe.”

For example, she said, “we work with payers to establish a reasonable reimbursement and explore ways of manufacturing our products more efficiently. These elements are more the challenge of a therapeutic developer than a patient advocacy group.”

Potential Benefits in Hemophilia, Sickle Cell Disease

More specifically, Lambert said ARM is excited about gene therapy’s “potential to tackle a wide variety of rare disorders” including hemophilia and sickle cell disease—especially following the success of onasemnogene abeparvovec-xioi (Zolgensma®), a gene therapy for spinal muscular atrophy (SMA).

“Yes, Zolgensma is expensive,” she said, referring to the one-time therapy’s $2.1 million retail cost. “Sometimes people are startled by the prices of some of these therapies. That’s understandable. But we try to think about the value that therapy brings. Absent some kind of treatment, a child with SMA type 1 is going to die at age 2, a rather difficult and miserable death. The fact we now have this therapy that can actually save this child’s life is a dramatic thing.”

She added: “We have actually found that the price of these therapies isn’t the limiting factor. Both public and private payers see the value in the therapy and have been willing to pay for it on behalf of their clients or subscribers.”

The situation is similar with hemophilia; traditional factor replacement therapies cost $100,000 to $200,000 a year.

“If you could cure that patient for $2 million, it could actually save the healthcare system money,” Lambert said. “We’d be happy to do that, but our priority is actually in making the patient’s life better—and we believe these therapies will do that.”

Brett Kopelan is executive director of DEBRA, a New York City-based nonprofit that advocates for patients with epidermolysis bullosa (EB), a rare and incurable genetic connective tissue disorder characterized by extremely fragile skin. The father of a 15-year-old girl with EB, also known as the “butterfly disease,” he’s also on the 29-member board of directors of ARM.

“What’s good for the cell and regenerative medicine space is good for rare disease patients,” Kopelan said by phone from his home in Boulder, Colorado. “So if policies are developed, and legislation enacted, that helps either from a regulatory or a reimbursement perspective, that will help rare disease patients in the long term. And that’s one of the reasons why I got involved.”

Kopelan, who’s been on the ARM board for 3 years, added that “bringing in the voice of the patient in drug development just makes drug development more effective.”

Reducing High Manufacturing and Development Costs

Also on ARM’s board is Debra Miller, founder and CEO of CureDuchenne, a nonprofit based in Newport Beach, California, that advocates for boys with Duchenne muscular dystrophy.

“We appreciate the incredible contributions that Janet Lambert made during her tenure and look forward to the leadership of Tim Hunt,” Miller said in an emailed statement. “Rare diseases, like Duchenne, need the support that ARM provides, so that we can accelerate development of therapies and access to novel treatments for patients.”

Of particular interest to ARM is the 1983 Orphan Drug Act, a provision enacted shortly after NORD’s founding that offers pharmaceutical manufacturers patent exclusivity and various tax breaks to entice them to develop drugs for rare conditions.

“The act created a whole special set of incentives that help it make economic sense for developers to address orphan diseases and many gene therapies,” Lambert said. “Our expectation is that it will be renewed. I would say we’re paying attention to it, but I wouldn’t say that we’re concerned at this point.”

Perhaps a bigger challenge is how to bring down the actual cost of manufacturing gene therapies. At the moment, she said, many efforts are underway to improve the capability, consistency, and affordability of both cell and gene therapies.

“For example, in the cell therapy space, which is currently being used to treat some rare cancers, the challenge is you’re extracting cells from a very sick patient, then genetically engineering them at a different location—then you’re returning them to the site of the patient.”

“Also, you have a lot of variability in the starter material, the cells themselves,” she added. “Because if the patient is not healthy, the cells are not very healthy. So there’s a lot of complexity in that whole process.”

These topics and others will be the focus of ARM’s annual gathering, dubbed the Cell & Gene Meeting on the Mesa, set for October 11-13, 2022, in Carlsbad, California. Some 1700 attendees are expected to participate in this year’s hybrid conference.

“Biology always throws us curveballs that we didn’t anticipate,” Lambert said. “This year could be actually a record‑breaking year, in terms of gene therapies for rare disease coming to market. We are very excited about moving from a world where we’re counting not 2 or 3 approvals a year, but where we’re seeing regulatory authorities around the world approve 10 or 15 new gene therapies—many of which will address rare disease—every year. But we’re going to get there.”